Department of Hematology and Medical Oncology, Winship Cancer Institute of Emory University, Atlanta, GA, USA.
Winship Cancer Institute of Emory University, 1365 Clifton Road NE, Atlanta, GA 30322, USA.
Blood Rev. 2018 Mar;32(2):89-95. doi: 10.1016/j.blre.2017.09.001. Epub 2017 Sep 18.
Acute promyelocytic leukemia (APL) is a hyper-acute illness and presents with profound cytopenias in most patients and disseminated intravascular coagulation (DIC). Excellent treatment options are now available with drugs such as all-trans retinoic acid (ATRA), arsenic trioxide (ATO), anthracyclines and cytarabine. The outcome in APL has improved tremendously in the last 50years due to better understanding of the disease, development of effective targeted agents and improvement in supportive care. Carefully selected groups of patients treated in large multi-center trials on a protocol and in experienced centers have shown survival rates in excess of 85%. However population data and other studies show that approximately 30% of patients die during induction. This is an Institutional, national and global problem and remains a pressing and frustrating challenge in APL. While most APL experts are aware of the high rate of early deaths (ED), such awareness is not typically present among general hematologists and oncologists. Our area of focus over the last 7years has been the reduction of ED in both academic and community centers; as a result we have acquired substantial experience in APL induction. Two centers have implemented population-wide prospective trials; Brazil and Georgia/South Carolina, USA with improvement in the ED rate. Both centers used standardized guidelines along with consultative support and sharing of expertise which proved effective and helped to decrease ED. Induction mortality in APL is 30% or greater. We believe ED is largely preventable and population-wide survival can be improved. An effective strategy is to utilize a set of simplified treatment guidelines coupled with support from a group of experts during induction. Treating oncologists in both academic and community hospitals should receive aggressive education about ED and be encouraged to seek advice from a core group of established APL experts. This model could be implemented nationally to improve population-wide survival in this most curable leukemia.
急性早幼粒细胞白血病（APL）是一种超急性疾病，大多数患者表现为严重的细胞减少症和弥散性血管内凝血（DIC）。由于对疾病的认识不断加深，以及有效靶向药物和支持性治疗的改进，目前有多种药物可供选择，如全反式维甲酸（ATRA）、三氧化二砷（ATO）、蒽环类药物和阿糖胞苷。在过去的 50 年中，由于对疾病的认识不断加深，开发了有效的靶向药物，并改善了支持性护理，APL 的治疗效果有了极大的改善。在方案和经验丰富的中心进行的大型多中心试验中，精心选择的患者组的生存率超过 85%。然而，人群数据和其他研究表明，大约 30%的患者在诱导期死亡。这是一个机构、国家和全球的问题，在 APL 中仍然是一个紧迫和令人沮丧的挑战。虽然大多数 APL 专家都意识到早期死亡率（ED）很高，但普通血液学家和肿瘤学家通常没有这种意识。在过去的 7 年中，我们的重点一直是减少学术和社区中心的 ED；因此，我们在 APL 诱导方面积累了丰富的经验。两个中心已经实施了全国范围的前瞻性试验；巴西和美国佐治亚州/南卡罗来纳州，ED 率有所改善。这两个中心都使用了标准化指南以及咨询支持和专业知识共享，事实证明这是有效的，并有助于降低 ED。APL 诱导死亡率为 30%或更高。我们相信 ED 在很大程度上是可以预防的，并且可以提高人群的生存率。一个有效的策略是利用一套简化的治疗指南，并在诱导期间得到一组专家的支持。学术和社区医院的肿瘤治疗医生应该接受关于 ED 的积极教育，并鼓励他们向一组成熟的 APL 专家寻求建议。这种模式可以在全国范围内实施，以提高这种最可治愈白血病的人群生存率。
